Sangamo Biosciences: Editing DNA to treat ALS and other diseases

An innovative zinc finger based technology is currently in clinical trials and holds promise for treating several diseases. Their name is derived from their 3D structure in which a zinc atom holds two loops of the protein together to form a “finger.” There are several therapeutic applications for zinc fingers, including its use for repairing a disrupted gene in patients. Sangamo Biosciences, a small company in Richmond, California, is largely responsible for the technique’s development and has licensed the technology to Sigma-Aldrich, who will make these agents available to the research community. Sangamo has reserved the right to develop medical applications of the technology for itself and has four Phase 2 clinical trials in progress, including study of SB-509 for the treatment of ALS.

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